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FDA issues final guidance about multiple endpoints in clinical trials

Today, FDA issued the Multiple Endpoints in Clinical Trials Final Guidance for Industry. This final guidance provides sponsors and review staff with the Agency’s thinking about the problems posed by multiple endpoints in the analysis and interpretation of study results and how these problems can be managed in clinical trials for drug development. It builds on the International Council for Harmonisation guidance for industry E9 Statistical Principles for Clinical Trials, which was released in September 1998. With this final guidance, FDA aims to provide greater detail on multiple endpoints beyond what was discussed in that document.

This guidance describes various strategies for grouping and ordering endpoints for analysis of a drug’s effects and applying some well-recognized statistical methods for managing multiplicity (the existence of many comparisons in a clinical trial which, without appropriate statistical adjustments, can lead to a higher-than-intended rate of making false conclusions about a drug’s effect) within a study to control the chance of making false conclusions about a drug’s effects. Most clinical trials performed in drug development contain multiple endpoints to assess the effects of the drug and to document the ability of the drug to favorably affect one or more disease characteristics. When more than one endpoint is analyzed in a single trial, the likelihood of making false conclusions about a drug’s effects with respect to one or more of those endpoints could increase if there are no appropriate adjustments for multiplicity. Basing a conclusion on an analysis where the risk of false conclusions has not been appropriately controlled can lead to false or misleading representations regarding a drug’s effects.

This guidance finalizes the draft guidance of the same title that FDA issued in 2017. Please read the Guidance Snapshot for an overview of the document and listen to the corresponding Guidance Recap Podcast with Dr. John Lawrence, a statistician in CDER’s Office of Biostatistics, for more highlights and background about the guidance.

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